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Gene therapies aim for the big goal of edits in vivo
The field of gene therapy is experiencing major advances driven by precise editing technologies, such as base and prime editing, and by the design of increasingly sophisticated vectors to deliver payloads that could reverse the effects of diseases. However, in the transition to in vivo applications many approaches still fail in their attempt to effectively reach target tissues or cells.

Gene therapies aim for the big goal of edits in vivo
The field of gene therapy is experiencing major advances driven by precise editing technologies, such as base and prime editing, and by the design of increasingly sophisticated vectors to deliver payloads that could reverse the effects of diseases. However, in the transition to in vivo applications many approaches still fail in their attempt to effectively reach target tissues or cells.
Mesothelin is biomarker, potential target in arthritic bone damage
In a recent study published in Cell Reports Medicine, researchers from the Institute of Chinese Materia Medica of the China Academy of Chinese Medical Sciences and Xi’an Jiao Tong University Health Science Center found that Mesothelin (MSLN) was significantly elevated in rheumatoid arthritis (RA) patients and collagen-induced arthritis (CIA) rats and mice, where it drove osteoclast differentiation.
Harmane-based quaternary ammonium salt against drug-resistant S. aureus
Researchers at Shandong Second Medical University have developed a dimethyl quaternary ammonium derivative of harmane that shows strong potential for attacking the bacterium through diverse mechanisms.
Opko’s OPK-88006 shows hepatoprotection in MASH
Opko Health Inc. has recently presented data for their GLP-1/glucagon receptor dual agonist OPK-88006, which is in preclinical development for the treatment of metabolic disease, including metabolic dysfunction-associated steatohepatitis (MASH) and obesity.
CDK7 sustains fibrolamellar carcinoma cell identity and survival
Fibrolamellar carcinoma (FLC) is a rare and lethal cancer accounting for 1% of liver cancers affecting young, otherwise healthy patients. The primary driver of the vast majority of human FLCs is a fusion between the heat shock protein 40 (DNAJB1) and the catalytic subunit α of protein kinase A (PRKACA).
Peptidream files INDs for [225Ac]PD-32766 and [64Cu]PD-32766
Peptidream Inc. has submitted IND applications to the FDA to initiate phase I studies of [225Ac]PD-32766, as a potential radiotherapeutic, and [64Cu]PD-32766, as the radiodiagnostic pair, for patients with clear cell renal cell carcinoma (ccRCC).
Toolgen and Geneditbio to advance genome-editing therapeutics
Toolgen Inc. has entered into a strategic cross-license agreement with Geneditbio Ltd. to jointly advance the development of next-generation in vivo genome-editing therapeutics.
Captor Therapeutics patents new Nek7 degradation inducers
Captor Therapeutics Inc. has disclosed molecular glue degraders comprising a cereblon (CRBN) binding moiety acting as serine/threonine-protein kinase Nek7 degradation inducers reported to be useful for the treatment of amyotrophic lateral sclerosis, Still’s disease, Behçet’s disease, familial Mediterranean fever, Alzheimer’s disease, pulmonary arterial hypertension and more.
Gemma’s GB-221 gains trial clearance in Brazil for SMA1
Gemma Biotherapeutics Inc.’s GB-221, a novel gene therapy for spinal muscular atrophy type 1 (SMA1), has received clinical trial clearance from ANVISA, Brazil’s health regulatory agency.

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